tbuckley/cabot-search huggingface dataset:
PB1996: CYTOGENETIC FEATURES OF 164 MOROCCAN PATIENTS WITH MYELODYSPLASTIC SYNDROME
Authors: Yossra Aghoutane, Maha EL Amani, Imane Ait el Filali, Moussa Bouh Hawa, Fatima Zahra Lbarrah, Othman Doghmi, Selim Jennane, El Mehdi Mahtat, Hicham El Maaroufi, Doghmi Kamal
Journal: HemaSphere (2023)

Topic: 9. Myelodysplastic syndromes - Biology & Translational Research Background: Cytogenetics is a crucial tool for the diagnosis, classification and prognostic stratification of myelodysplastic syndromes (MDS). Conventional cytogenetics (CC) (karyotyping) of bone marrow cells remains the gold standard in detecting MDS-associated cytogenetic abnormalities. Few data on the cytogenetic features of MDS in Moroccan patients are reported in the literature. Aims: Describe the cytogenetic features of patients diagnosed with myelodysplastic syndrome treated in our center. Methods: In this descriptive retrospective study, we enrolled 164 patients diagnosed with myelodysplastic syndrome and treated in the department of clinical hematology at Mohammed V Military Hospital in Rabat/Morocco, between January 2009 and December 2022. Results: 164 patients diagnosed with myelodysplastic syndrome were included. Median age was 67 years with a male predominance of 64% [sex ratio: 1.8]. The cytogenetic analysis failed in 3 patients (1.8%) and detected clonal cytogenetic abnormalities in 52 patients (31.7%). Prognostic group distribution according to the IPSS-R was found to be very good in 5 patients (3%) with 4 cases of loss of chromosome Y and 1 case of 11q deletion. The good cytogenetic group was predominant (68.9%), 109 patients (66.5%) had normal karyotype, 1 case of 5q deletion associated with trisomy 8, 1 case of 20q deletion, 1 case of 5q deletion and 1 case of chromosome 20 loss. The prognosis was intermediate in 31 patients (18.9%), 14 patients had a single chromosomal abnormality, 10 had double chromosomal abnormality, 6 cases of trisomy 8 and 1 case of 7q deletion. Five patients (3%) had a poor prognosis including 3 patients with complex karyotype with 3 chromosomal abnormalities and 2 patients with monosomy 7. The prognosis was very poor in 7 patients (4.3%) who had complex karyotype with more than 3 chromosomal abnormalities. Summary/Conclusion: The landscape of karyotype abnormalities in our population revealed a predominance of the good prognosis group with a normal karyotype, hence the need of molecular biology, in particular the MDS NGS panel, for the elaboration of a molecular score in order to refine the prognostic stratification of our patients. Keywords: MDS
PB2360: HIV-ASSOCIATED BURKITT LYMPHOMA IN THE COMBINATION ANTIRETROVIRAL THERAPY ERA: REAL-WORLD OUTCOMES AND PROGNOSTICATION
Authors: Chaoyu Wang, Jieping Liu, Yao Liu
Journal: HemaSphere (2023)

Topic: 19. Aggressive Non-Hodgkin lymphoma - Clinical Background: Little is known about the incidence, clinicopathologic characteristics, prognostic factors, and survival of Burkitt’s lymphoma in people with HIV as these are less common than HIV-negative lymphoma, and currently, there are no standard guidelines for treatment of these patients. Aims: All newly diagnosed patients with HIV-associated BL between March 2012 and February 2022 at CUCH, were retrospectively reviewed. Methods: We performed a retrospectively study to analyze the clinical characteristics and outcomes of HIV-associated Burkitt’s lymphoma in Chongqing University Cancer Hospital (CUCH), southwest China, from March 2012 to February 2022. Results: In the entire cohort, the median age was 36 years (range, 28–60 years), and more patients were male (82.4%). The median CD4+ T cell count was 214/μl (range, 54-601), of whom 47.1% had a CD4+ T cell count below 200/μl. Most patients had elevated LDH, elevated β2-MG, extranodal involvement and advanced Ann Arbor stage at diagnosis. With a median follow-up of 11.5 months (range, 1.6-94.9 months), the overall 1-year PFS and OS rates were 27.6% and 47.6% respectively. The 1-year OS times in the LDH<3 upper limit of normal and LDH≥3 upper limit of normal groups were 62.5% and 31.3%, respectively (p=0.008). The 1-year OS times in the received <4 cycles and ≥4 cycles groups were 0 and 77.8%, respectively (p<0.001). Summary/Conclusion: These results demonstrated that LDH<3 upper limit of normal and received ≥4 cycles chemotherapy were significantly associated with improved outcomes. However, rituximab administration was not significantly associated with improved outcomes.Keywords: Burkitt’s lymphoma, HIV
PB2425: EFFICACY AND SAFETY OF COMBINED THERAPY OF RECOMBINANT HUMAN THROMBOPOIETIN AND ELTROMBOPAG FOR TREATMENT OF PERSISTENT THROMBOCYTOPENIA AFTER HEMATOPOIETIC STEM CELL TRANSPLANTATION
Authors: Pan Pan, Aiming Pang
Journal: HemaSphere (2023)

Topic: 22. Stem cell transplantation - Clinical Background: Persistent thrombocytopenia (PT) is a critical complication after allogenic hematopoietic stem cell transplantation (allo-HSCT) with a incidence ranging from 20%-40%, associated with high risk thrombocytopenic bleeding and poor prognosis for patients undergoing allo-HSCT. However, the specific factors is remaining elusive and the standard treatment is yet to manage. Aims: To evaluate the efficacy and safety of the combined therapy of recombinant human thrombopoietin (rhTPO) and eltrombopag (EPAG) for the patients with PT after allo-HSCT. Methods: This study retrospectively analyzed 20 patients who received continuous rhTPO 15000u Qd iH and EPAG 50mg Qd po for the treatment of PT. Results: A total of 13 (65%) patients responded to rhTPO treatment at a median time of 10 days (range 4-46) after initiation of combined therapy (Fig 1). Megakaryocytes in bone marrow of responders were significantly higher than that of non-responders (10 vs 3.5; P=0.037). Importantly, combined therapy was well tolerated in all but one patient who quit the trial, there was no evidence of grade 3–4 toxicities till last follow-up. In the Kaplan-Meier survival analysis, 1-year probabilities of overall survival (100% [95% CI, 100–100%] VS 42.9% [95% CI, 18.2–100%]; p=0.0021) and disease-free survival (92.3% [95% CI,79.3-100%] VS 28.6% [95% CI,8.9-92.2%], P=0.0027) of responders was significantly higher than that of non-responders respectively. Fig.1 The cumulative incidence of overall response (65%, n=20). (Created by R statistical software 4.2.2 (R Foundation for Statistical Computing, Vienna, Austria).)Summary/Conclusion: The results of this study indicate that combined therapy of rhTPO and eltrombopag might be a safe and effective treatment for PT after HSCT, even the combined therapy could improve prognosis of patients undergoing PT after HSCT. Keywords: Hematopoietic cell transplantation, Thrombopoietin (TPO), Platelet, Thrombocytopenia
PB2439: EARLIER ENGRAFTMENT WITHOUT INCREASE OF GVHD IN HIGHER DOSE OF CD34+ CELLS IN HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION
Authors: Tran-Der Tan, Lun-Wei Chiou, Rong-Long Chen
Journal: HemaSphere (2023)

Topic: 22. Stem cell transplantation - Clinical Background: Haploidentical hematopoietic stem cell transplantation is the alternative source of donor selection for allogeneic transplants when matched related or unrelated donors are unavailable. However, compared with the matched related or unrelated transplants, haploidentical transplants may have a higher risk of graft-vs-host disease (GVHD) and delayed engraftment of neutrophils. The former has been overcome by the post-transplant high-dose cyclophosphamide (PTCY), and the latter is still to be a concern. Aims: We tried to infuse a higher cell dose of haploidentical hematopoietic stem cells and observe the period of neutrophil recovery and the incidence of acute and chronic GVHD. Methods: We harvested a higher amount of peripheral blood hematopoietic stem cells from haploidentical donors and compared the period of neutrophil recovery between the CD34+ cell counts higher or lower than 10 million/kg of the patient’s body weight. Results: Twenty-six patients have undergone twenty-seven transplants at our institute between October 2014 and March 2021. The median age of our patients was 35, with a range between 5 and 64, including 14 males and 12 females. Our cohort consisted of eight AMLs, four ALLs, 1 ATLL, and 1 CLL with Richter transformation, 3 HL, 3 NHL, and 6 AA. We have two to five mismatches in ten loci, and the median CD34+ cells harvested were 8.7 x 10e6/kg (ranging between 2.2 and 16.8) with more than 10 x 10e6/kg of CD34+ cells in 10 patients, and less than 10 x 10e6/kg of CD34+ cells harvested in 16 patients. The conditioning regimens were myeloablative in eight and reduced intensity in nineteen patients. The median engraftment days were 19 versus 14 days in CD34+ cells below and higher than 10 x 10e6/kg. The median time of platelet count above 20,000/ul and transfusion-independent were 16 versus 10 days in CD34+ cells below and higher than 10 x 10e6/kg. There was no difference in acute and chronic GVHD incidences between total nuclear cells (TNC) above or lower 1.2 x 10e8/kg and CD34+ cells above or lower 10 x 10e6/kg harvest of cells. Summary/Conclusion: The patients receiving more CD34+ stem cells have faster engraftment of neutrophils and platelets, and there was no significant difference in the incidences of acute and chronic GVHD.Keywords: Engraftment, Allogeneic stem cell transplant, Graft-versus-host disease (GVHD), Haploidentical stem cell transplantation
PB2049: THE EFFICACY OF HETROMBOPAG PLUS CYCLOSPORINE A IN PATIENTS WITH TRANSFUSION-DEPENDENT NON-SEVERE APLASTIC ANEMIA: A PROSPECTIVE REGISTRY STUDY
Authors: Yuemin Gong, Xiaoyu Chen, Xinyu Zhang, Guangsheng He, Jianyong LI
Journal: HemaSphere (2023)

Topic: 12. Bone marrow failure syndromes incl. PNH - Clinical Background: The therapeutic approach for transfusion-dependent non-severe aplastic anemia (TD-NSAA) is undetermined. Though the British guideline recommended intensive immunosuppressive therapy as the first line therapy for TD-NSAA, the cost and risk were relatively high. Hetrombopag, a thrombopoietin receptor agonist (TPORA), has shown encouraging results in severe aplastic anemia, but its efficacy has not been investigated in TD-NSAA. Aims: This study aimed to investigate the efficacy of hetrombopag plus cyclosporine A (CsA) for TD-NSAA. Methods: Hetrombopag was taken at an initial dose of 7.5 mg per day to a maximum of 15 mg per day. CsA was basically initiated at 3~5 mg/kg daily and then adjusted according to the response or side effects, to maintain the target concentration between 150 and 250 μg/L. The primary endpoint was hematologic response in ≥ 1 lineage. Results: 19 eligible patients who were enrolled and received hetrombopag plus CsA from September 2021 to February 2023 were collected by prospective registration in the Chinese Eastern Collaboration Group of Anemia (ChiCTR2100045895) (Table 1). The median follow-up period was 8.2 [0.8~16.8] months. 14 of the 19 patients (74%) responded in ≥ 1 lineage, including 13 patients (68%) achieving partial response and one patient (5%) achieving complete response. The median time to initial hematologic response was 6.1 [1.7~28.4] weeks. 9 of 15 patients (60%) achieved erythroid response; 13 of 19 patients (68%) achieved platelet response and 11 of 14 patients (79%) achieved neutrophil response. The median time to erythroid, platelet and neutrophil response were 15, 8.3 and 19.7 weeks, respectively. The survival rate was 95%, with one patient died of intracerebral hemorrhage 9 months after initiation of hetrombopag. Drug-induced hepatic or renal injury happened in 4 patients (21%); all showed remission after dose reduction or discontinuation. Summary/Conclusion: The combination of hetrombopag with CsA is an effective regimen in patients with TD-NSAA. Table 1 Baseline Clinical Characteristics of Patients - Characteristic Follow-up — months 8.2 [Range: 0.8~16.8] Age — yrs 50 [Range: 18-67] Age category — no. (%) <40 6 (32) ≧40 13 (68) Sex — no. (%) Female 10 (53) Male 9 (47) Transfusion dependence category — no. (%) RBC dependent 15 (79) Platelet dependent 19 (100) Laboratory values Hemoglobin — g/L 85 [IQR: 67-101] Neutrophil count — per μL 1030 [IQR:818-1600] Lymphocyte count — per μL 1675 [IQR: 1105-2365] Platelet count — per μL 20500 [IQR: 10000-33750] Reticulocyte count — per μL 54700 [IQR: 34400-71000] RDW-CV % 14.5 [IQR: 13.3-18.9] Keywords: Aplastic anemia, Thrombopoietin (TPO)
PB2472: HOSPITAL BURDEN OF VENO-OCCLUSIVE DISEASE (VOD): COMPARISON OF HOSPITAL STAYS WITH VOD PRINCIPAL VERSUS SECONDARY DIAGNOSIS
Authors: Saurabh Aggarwal, Sushil Kumar, Ozlem Topaloglu
Journal: HemaSphere (2023)

Topic: 22. Stem cell transplantation - Clinical Background: Veno-occlusive disease (VOD), also called sinusoidal obstruction syndrome (SOS), is a potentially life-threatening complication of hematopoietic stem cell transplantation (HSCT) conditioning or high-dose nontransplant chemotherapy. Aims: To compare hospitalizations with principal and secondary diagnosis of Veno-Occlusive Disease (VOD). Methods: The National Inpatient Sample (NIS) data set from the Healthcare Cost and Utilization Project was utilized in order to analyze hospital admission records for patients with VOD. Three cohorts of patients were designed: Any diagnosis, principal and secondary. Thirty comorbidities were assessed using Elixhauser scoring. Multivariate logistic regression was conducted to assess predictor variables for LOS and costs. Results: In 2016, there were an estimated 200 hospitalizations with a diagnosis of VOD, with 35 as principal diagnosis and 165 as secondary. The mean ages in three groups were: 21.1 (SD 21.97), 15.29 (SD 21.48) and 22.33 (SD 22.2) years for any, principal and secondary diagnosis groups, respectively. Nearly half of all VOD patients were covered by Medicaid (48%-Any, 29%-Principal, 52%-Secondary). Majority of patients in each group were white (43%-Any, 57%-Principal,39%-Secondary). Interestingly, the mean length of stay was significantly higher in patients with secondary diagnosis (45.35-Any, 20.71-Principal, 50.57-Secondary, p<0.05). The mean hospital charges were also ~3 times higher in the secondary diagnosis group ($1,152,370-Any, $398,930-Principal, $1,312,191-Secondary, p<0.05). The top leading non-VOD principal diagnoses were Acute lymphoblastic leukemia and Acute kidney failure. Summary/Conclusion: OD is a serious disorder with very long LOS and high hospital costs. This shows that patients with secondary diagnosis of VOD incur higher costs than those with principal diagnosis. Keywords: Veno-occlusive disease
PB2131: MAGNETISMM-7: AN OPEN-LABEL, MULTICENTER, RANDOMIZED PHASE 3 STUDY OF ELRANATAMAB VERSUS LENALIDOMIDE IN POST-TRANSPLANT PATIENTS WITH NEWLY DIAGNOSED MULTIPLE MYELOMA
Authors: Maria Victoria Mateos Manteca, Sebastian Grosicki, Kihyun Kim, Eric Negre, Erik Vandendries
Journal: HemaSphere (2023)

Topic: 14. Myeloma and other monoclonal gammopathies - Clinical Background: Elranatamab is a humanized bispecific antibody targeting B cell maturation antigen (BCMA) on myeloma cells and CD3 on T cells. Elranatamab has shown promising efficacy and acceptable safety in patients with relapsed and/or refractory multiple myeloma (MM). Aims: The aim of the MagnetisMM-7 study is to evaluate elranatamab monotherapy vs lenalidomide monotherapy in patients with newly diagnosed MM (NDMM) after undergoing autologous stem cell transplant (ASCT). Methods: MagnetisMM-7 is an ongoing, open-label, 2-arm, multicenter, randomized phase 3 study estimated to enroll ~700 patients. ClinicalTrials.gov ID: NCT05317416. Patients are randomized to receive either subcutaneous elranatamab or oral lenalidomide once daily. After initial step-up dosing, two alternative dosing regimens of elranatamab are examined. The primary endpoint is progression-free survival (PFS) assessed by blinded independent review per International Myeloma Working Group (IMWG) criteria (up to ~5 y). Secondary endpoints include overall survival, minimal residual disease (MRD) negativity rate at 12 mo after randomization per IMWG criteria as assessed via next-generation sequencing (NGS), sustained MRD negativity rate at 24 mo after randomization as assessed via NGS, PFS by investigator, duration of MRD negativity, complete response (CR) rate, duration of CR, safety, quality of life, immunogenicity, and pharmacokinetics. Key inclusion criteria are: ≥18 y; a diagnosis of MM with measurable disease according to IMWG criteria; history of induction therapy for NDMM, followed by high dose therapy and ASCT with or without consolidation; partial response or better according to IMWG criteria; identification of the dominant malignant clone; and Eastern Cooperative Oncology Group performance status ≤1. Randomization must occur ≤120 d from ASCT; for patients who receive consolidation therapy after ASCT, randomization must occur ≤60 d of consolidation and ≤7 mo of ASCT. Key exclusion criteria are: plasma cell leukemia; amyloidosis; Waldenström’s macroglobulinemia; POEMS syndrome; known active CNS involvement or clinical signs of myelomatous meningeal involvement; previous MM maintenance treatment; prior treatment with BCMA-targeted therapy; any other active malignancy ≤3 y of enrollment; active, uncontrolled bacterial, fungal, or viral infection; and previous administration of an investigational drug or vaccine ≤30 d or 5 half-lives preceding the first dose of study treatment. As of February 2023, the study is open and enrolling at 29 sites in 13 countries. Results: N/A Keywords: Clinical trial, Bispecific, Multiple myeloma, B-cell maturation antigen
PB2399: BRENTUXIMAB VEDOTIN IN TREATING CHINESE PATIENTS WITH LYMPHOMA:A MULTICENTER,REAL-WORLD STUDY
Authors: Xudong Zhang, Honghan Qiao, Xiaofei Chai, Xue Gao, Rongjun MA, Yufu LI, Zunmin Zhu, Mingzhi Zhang
Journal: HemaSphere (2023)

Topic: 20. Lymphoma Biology & Translational Research Background: Brentuximab vedotin (BV) was approved as a therapy for patients with CD30-positive lymphoma in China in 2020 based on the results of multiple clinical trails.Few Chinese real-world data of its use are yet available. Aims: To reflect the application situation of brentuximab vedotin in patients with lymphoma among different hospitals in Henan province in China under real-world conditions. Methods: This was a multicenter and retrospective study in 104 patients with lymphoma who received brentuximab vedotin for the first time between August 2020 and September 2022 across eight centers in Henan province.Data on the clinical use,effectiveness and adverse events (AEs) of brentuximab vedotin were extracted from patient medical records.Short-term effectiveness was assessed based on objective response rate(ORR),complete response(CR),partial response(PR),stable disease(SD) and progressive disease(PD).Progression-free survival(PFS) and overall survival(OS) were calculated using Kaplan-Meier method.Safety was also evaluated in our study. Results: 104 lymphoma patients were enrolled in our study,who had completed a median of 2 cycles(range,1-8) of BV-based treatment.A total of 72.1% of patients were relapsed/refractory lymphoma,and only 27.9% were previously untreated lymphoma who received BV in frontline treatment settings.Among them all,the ORR achieved 64.5%(CR 25.8%,PR 38.7%).After a median follow-up of 10.2 months,the 6-month PFS rate and OS rate achieved 79.1% and 88.1% respectively,and the 12-month PFS rate and OS rate achieved 79.1% and 79.8% respectively.In general,BV-based treatment was well-tolerated,with 38.5% incidence of grade≥3 AEs.The most commonly reported AEs were hematologic disorders,especially neutropenia,with the incidence reaching 50.0%. Summary/Conclusion: Brentuximab vedotin-based regimens could be a promising therapeutic option with remarkable effectiveness and moderate toxicity in treating Chinese lymphoma patients with CD30 expression. Keywords: CD30, Real world data, Lymphoma
PB2674: CLINICAL, NEUROIMAGING, AND BEHAVIORAL RISK FACTORS FOR NEUROCOGNITIVE IMPAIRMENT IN CHINESE PATIENTS WITH HEMOPHILIA: A MULTICENTER STUDY
Authors: Yin Ting Cheung, Chung Tin Justin MA, Henry Hon Wai Lam, Siu Cheung Ling, Kevin Kwok, Chak Ho LI, Chung Yin Ha, Sze Fai Yip, Raymond Wong, Winnie Chiu Wing Chu, Chi Kong LI
Journal: HemaSphere (2023)

Topic: 35. Quality of life and palliative care Background: Despite a lack of clinical symptoms, subclinical microstructural changes in the brain might contribute to cognitive impairment in patients with hemophilia. Few studies have evaluated the impact of neuroimaging and psychosocial factors on cognitive function. Aims: To determine the prevalence and characteristics of cognitive impairment in patients with hemophilia, and identify associated risk factors in this population. Methods: We recruited hemophilia A or B patients who were aged ≥ 10 years old from three public hospitals in Hong Kong. A neurocognitive battery was administered to evaluate their attention, memory, processing speed and cognitive flexibility performances. They also underwent magnetic resonance imaging to identify cerebral microbleeds. Validated self-reported questionnaires were administered to assess their mental health status and adherence to prophylactic treatment. General linear modeling was used to investigate the association of neurocognitive outcomes with risks factors, adjusting for age and education attainment. Results: Forty-two patients were recruited (median age 32.0 years; 78.6% hemophilia A; 80.9% moderate-to-severe disease severity). Six patients (14.3%) had developed cerebral microbleeds. As a cohort, the mean cognitive scores of patients with hemophilia were significantly worse than the population norms on multiple cognitive domains (Figure 1), A subgroup of patients demonstrated impairments in cognitive flexibility (50%), motor processing speed (47.6%), and attention (19%). Hemarthrosis in the previous year was associated with worse attention (Estimate=7.62, standard error [SE]=3.81; p=0.049) and cognitive flexibility (Estimate=8.64, SE=5.84; p=0.043). Compared to patients with a normal weight, patients who were overweight/obese had worse cognitive flexibility (p=0.026). Depressive (Estimate=0.22, SE=0.21; p=0.023) and anxiety (Estimate=0.26, SE=0.07; p=0.0069) symptoms were associated with inattentiveness. Among patients who were receiving prophylactic treatment (71.4%), medication adherence was positively correlated with cognitive flexibility (p=0.037). Summary/Conclusion: A substantial proportion of patients with hemophilia demonstrated cognitive impairment, particularly higher-order thinking skills. Screening for cognitive deficits should be incorporated into routine care. Future studies should evaluate the association of neurocognitive outcomes with functional capacity and occupational/vocational outcomes.Keywords: Cerebral hemorrhage, Quality of life, Magnetic resonance imaging, Hemophilia